Diseases
Diseases in Gene-Editing Clinical Trials
Conditions where gene editing is being tested in people, as distinct from being discussed.
How this list is built
These are conditions where editing has reached actual patients in registered trials. It is a much longer list than the approved one and a much shorter list than the diseases people expect CRISPR to solve.
| # | Entry | Category | Status |
|---|---|---|---|
| 1 | Sickle Cell Disease An inherited blood disorder in which a single DNA letter change makes red blood cells… | Blood disorders | Approved |
| 2 | Beta Thalassemia An inherited disorder in which the body makes too little beta-globin, leaving many… | Blood disorders | Approved |
| 3 | ATTR Amyloidosis A disease in which a misfolded liver protein accumulates in the heart and nerves — and… | Metabolic disease | Phase III |
| 4 | Hereditary Angioedema A rare disorder causing sudden, unpredictable and sometimes life-threatening swelling… | Rare genetic disease | Phase III |
| 5 | High Cholesterol and Familial Hypercholesterolemia The most common cause of premature heart disease, and the first attempt to treat an… | Cardiovascular disease | Phase II |
| 6 | Cancer Not one disease but hundreds — and the place where gene editing is used to engineer… | Cancer | Phase II |
| 7 | Leukemia and Lymphoma Blood cancers, where engineered cell therapy has worked best — and where the first… | Cancer | Phase II |
| 8 | HIV A virus that hides inside the genome of the cells it infects — which is why editing has… | Infectious disease | Phase I |
| 9 | Duchenne Muscular Dystrophy A severe muscle-wasting disease caused by mutations in the largest gene in the human… | Muscular disease | Preclinical |
| 10 | Cystic Fibrosis A disease of thick mucus in the lungs and gut where highly effective drugs already exist… | Rare genetic disease | Preclinical |
| 11 | Hemophilia An inherited bleeding disorder in which gene addition reached approval first, leaving… | Blood disorders | Preclinical |
| 12 | Inherited Blindness Inherited retinal disease was where in vivo CRISPR was first injected directly into a… | Eye disease | Phase I/II |
| 13 | Alpha-1 Antitrypsin Deficiency A single-letter mutation that damages the lungs by absence and the liver by accumulation… | Rare genetic disease | Phase III |
| 14 | Chronic Granulomatous Disease A rare immune disorder in which white blood cells cannot kill the bacteria they engulf —… | Rare genetic disease | Phase I/II |
| 15 | Huntington's Disease A fatal inherited brain disease caused by a toxic protein — where the genetics are… | Neurological disease | Preclinical |
| 16 | Parkinson's Disease A common neurodegenerative disease that is mostly not genetic — which makes it a poor fit… | Neurological disease | Discovery |
| 17 | Alzheimer's Disease The most common cause of dementia, where a well-known risk gene exists but editing… | Neurological disease | Discovery |
| 18 | Diabetes Where editing is being used not to correct a gene but to hide transplanted… | Metabolic disease | Phase I |
| 19 | Retinitis Pigmentosa A group of inherited retinal degenerations with over 80 causative genes — where the… | Eye disease | Preclinical |
What this list does not tell youInclusion means research is happening, not that a treatment works or is available. Each disease page states its evidence level.